Fortress Biotech and Cyprium Therapeutics Announce $4.1 Million Grant from NINDS to Further Development of AAV-ATP7A Gene Therapy for Menkes Disease
MIAMI, March 04, 2024 (GLOBE NEWSWIRE) -- Cyprium Therapeutics, Inc. (“Cyprium”), a majority-owned subsidiary of Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”), today announced that the National Institute of Neurological Disorders and Stroke (“NINDS”) of the National Institutes of Health (“NIH”) has awarded a three-year grant totaling approximately $4.1 million to the Research Institute at Nationwide Children’s Hospital and Principal Investigator, Stephen G. Kaler, M.D., M.P.H., to fund completion of preclinical studies, manufacturing and preparation of an Investigational New Drug Application for a first-in-human clinical trial to advance adeno-associated virus (“AAV”)-ATP7A gene therapy, also known as AAV-ATP7A, for the treatment of Menkes disease.
- Often lethal if untreated, Menkes disease is an X-linked recessive disorder of copper metabolism caused by mutations in ATP7A, an evolutionarily conserved copper-transporting ATPase.
- “By combining CUTX-101 with working copies of ATP7A delivered by AAV, we hope to enhance clinical outcomes in Menkes disease, a fatal rare pediatric disease.
- Preclinical studies have demonstrated a synergistic effect of AAV-ATP7A and CUTX-101 in a reliable mouse model of Menkes disease.
- In early studies, cerebrospinal fluid (“CSF”)-directed AAV gene therapy rescued 22-53% of mice with a mutation in the human Menkes disease homolog (mottled-brindled) when combined with CSF or subcutaneous copper.